guidebook. - Fanconi Anemia Research Fund

Chapter 9: Matched Sibling Donor HSCT
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transplant physician’s recommendation; and (4) the
parental or adult patient’s decision.
The decision-making process in the timing of transplantation
is difficult and must include multiple factors:
• The vast majority of patients will progress to
aplastic anemia and/or MDS/AML without
transplant.
• Transplants for FA using matched sibling donors
have a very good chance of success, at 85-90%
in FA-specialized transplant centers;
• However, transplants are associated with a risk
of peritransplant mortality of 10-15% and a
risk of chronic GvHD for a “minimum” of 12%
(with unmodified transplants);
• In general, results of transplants are better for
patients with aplastic anemia than with MDS/
AML;
• Results of transplants are generally better for
patients who are younger, partly due to a lower
risk of GvHD; and
• The patient’s overall vital organ status, such as
renal or hepatic function, influences the transplant
outcome.
In addition to these factors, the following are relative
and absolute indications for transplantation of FA
patients from matched sibling donors based on patients’
hematologic status and age:
Absolute indications
• Severe aplastic anemia and transfusion dependence.
In this case, no trial of androgens prior to
proceeding to BMT.